Personalized Prediction of Tolerance and Immunogenicity in Hemophilia

NCT01626105 | Unknown

• 1 other identifier: PPTIH No. 1
• Study Type: observational
• Target: 55
• Locations: 1 country
• Sites: 1

Brief Summary

This study is designed to accurately identify the pharmacogenetic determinants of risk of Factor VIII (FVIII) inhibitor development by focusing on only a select group of Hemophilia A (HA) patients who have: (i) received a recombinant FVIII therapeutic product containing the same primary amino acid sequence since their original diagnosis; (ii) verifiable FVIII infusion histories; and (iii) been tested regularly for FVIII inhibitor development.

Conditions

Hemophilia A

Keywords

Hemophilia A; Factor VIII; Inhibitors; Immunogenicity; Pharmacogenetics; Mutation; Nonsynonymous Single Nucleotide Polymorphisms; Class II Human Leukocyte Antigens

Eligibility Criteria

• Age: 2 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Fifty five patients with severe or moderately severe hemophilia A who have received replacement therapy with a Factor VIII product representing only a single primary amino acid sequence.

You may qualify if:

• Patients with severe or moderately severe hemophilia A (HA) who have since birth been treated with only a single Factor VIII product (i.e., FVIII protein molecules containing only one primary amino acid sequence).

You may not qualify if:

• HA patients with severities other than severe or moderately severe.
• Hemophilia B patients.
• HA patients who have been treated with more than one FVIII product.
• HA patients who have been treated with more than one FVIII product.
• HA patients who do not have verifiable infusion histories.
• HA patients who lack documentable inhibitor testing \& infusion histories.

Sponsors & Collaborators

• Victor J Marder, M.D.: lead
• Baxter Healthcare Corporation: collaborator

Study Sites (1)

Children's Hospital of Michigan

Detroit, Michigan, 48201-2196, United States

Location

Related Publications (3)

• Yanover C, Jain N, Pierce G, Howard TE, Sauna ZE. Pharmacogenetics and the immunogenicity of protein therapeutics. Nat Biotechnol. 2011 Oct 13;29(10):870-3. doi: 10.1038/nbt.2002. No abstract available.

  PMID: 21997623 BACKGROUND

• Howard TE, Yanover C, Mahlangu J, Krause A, Viel KR, Kasper CK, Pratt KP. Haemophilia management: time to get personal? Haemophilia. 2011 Sep;17(5):721-8. doi: 10.1111/j.1365-2516.2011.02517.x. Epub 2011 Jun 8.

  PMID: 21649795 BACKGROUND

• Viel KR, Ameri A, Abshire TC, Iyer RV, Watts RG, Lutcher C, Channell C, Cole SA, Fernstrom KM, Nakaya S, Kasper CK, Thompson AR, Almasy L, Howard TE. Inhibitors of factor VIII in black patients with hemophilia. N Engl J Med. 2009 Apr 16;360(16):1618-27. doi: 10.1056/NEJMoa075760.

  PMID: 19369668 BACKGROUND

Related Links

• The Keck School of Medicine of USC
• The Los Angeles Orthopaedic Hospital in Alliance with UCLA Health System

Biospecimen

Retention: SAMPLES WITH DNA

A core laboratory exists for this study and will house a Biorepository containing the following: 1. White blood cells 2. Blood plasma

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Victor J. Marder, M.D.

  The Los Angeles Orthopaedic Hospital and The David Geffen School of Medicine at UCLA

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Tom E. Howard, M.D., Ph.D.

CONTACT

404-597-8014; thoward@txbiomedgenetics.org

Victor J Marder, M.D.

CONTACT

310-794-1663; vmarder@mednet.ucla.edu

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Associate Professor, Director Hemostasis, Pathology, Veterans Affairs Greater Los Angeles

Study Record Dates

• First Submitted: June 20, 2012
• First Posted: June 22, 2012
• Study Start: June 1, 2012
• Primary Completion: April 1, 2013
• Study Completion: June 1, 2014
• Last Updated: July 26, 2012

Record last verified: 2012-07

Locations

US (1)